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Research Highlight: Ozdinler and Silverman

ALS neuron damage reversed with new compound

Professors Hande Ozdinler and Rick Silverman have identified the first compound that eliminates the ongoing degeneration of upper motor neurons that become diseased and are a key contributor to ALS (amyotrophic lateral sclerosis), a swift and fatal neurodegenerative disease that paralyzes its victims.

In addition to ALS, upper motor neuron degeneration also results in other motor neuron diseases, such as hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). In ALS, movement-initiating nerve cells in the brain (upper motor neurons) and muscle-controlling nerve cells in the spinal cord (lower motor neurons) die. The disease results in rapidly progressing paralysis and death. So far, there has been no drug or treatment for the brain component of ALS, and no drug for HSP and PLS patients.

“Even though the upper motor neurons are responsible for the initiation and modulation of movement, and their degeneration is an early event in ALS, so far there has been no treatment option to improve their health,” said senior author Hande Ozdinler, associate professor of neurology at Northwestern University Feinberg School of Medicine. “We have identified the first compound that improves the health of upper motor neurons that become diseased.”

“I am very excited to find out if our hypothesis that stabilizing upper motor neurons in mice will translate to humans and NU-9 will provide hope for those inflicted with currently untreatable upper motor neuron diseases,” Silverman said. 

The research was published in Clinical and Translational Medicine

For more on the story read Northwestern Now

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